CAMBRIDGE, Mass., Sept. 28 /PRNewswire-FirstCall/ -- Transkaryotic
Therapies, Inc. (Nasdaq: TKTX) today announced that it has received a
Development Grant from the FDA's Office of Orphan Products Development (OOPD)
for iduronate-2-sulfatase (I2S), TKT's investigational enzyme replacement
therapy for the treatment of Hunter syndrome, also known as MPS II, a rare,
fatal disease. The $300,000 grant will pay for some costs of the company's
ongoing pivotal trial.
"We are grateful for this recognition of the importance of our efforts to
develop I2S as a potential treatment for Hunter syndrome. We fully support
the FDA's mission to encourage clinical development of products for rare
diseases and to put agency resources behind these development efforts," said
Michael J. Astrue, President and Chief Executive Officer of TKT.
Since 1989, approximately 36 products supported by OOPD grants have
received marketing approval by the U.S. Food and Drug Administration.
Clinical studies conducted under an Investigational New Drug application for a
rare disease qualify for consideration by the program. Grants of this size
are typically awarded to drugs in Phase III clinical trials.
The I2S pivotal trial, referred to as the AIM (Assessment of I2S in MPS
II) study, is a fully enrolled trial designed to evaluate safety and efficacy
of weekly and every-other week infusions of I2S administered at a dose of 0.5
mg/kg. Patients will receive a total of fifty-two infusions of either I2S
(patients randomized to the weekly dosing regimen), I2S alternating with
placebo (patients randomized to the every other week regimen), or placebo.
The AIM study is a twelve-month, randomized, double-blind, placebo-controlled
trial being conducted at nine sites around the world. The primary efficacy
endpoint in the trial is a single composite variable which combines two
clinical measurements: forced vital capacity as a measure of respiratory
function and the six-minute walk test as a measure of functional capacity.
Additional efficacy endpoints include measurements of joint range of motion
and combined liver/spleen size. TKT expects top-line results from the AIM
study in the second quarter of 2005 and, if positive, the company expects to
submit applications for marketing approval in both the United States and
Europe in the second half of 2005.
About I2S
I2S is a human iduronate-2-sulfatase produced by genetic engineering
technology intended for the long-term treatment of Hunter syndrome. The
rationale for the therapy is that I2S would replace enzyme that is deficient
in patients with Hunter syndrome and either stop or reverse disease
progression. I2S, the only known enzyme replacement therapy in development
for the treatment of Hunter syndrome, has been designated an orphan drug in
both the United States and Europe. In addition, I2S received Fast Track
designation from the FDA in July 2004. A Phase I/II clinical trial in 12
patients was completed in 2002 and all of those patients have remained on I2S
as part of an extension study designed to collect long-term safety data.
About Hunter Syndrome
Hunter syndrome is a chromosomally-linked genetic disorder affecting
males, also referred to as MPS II. This hereditary disorder is characterized
by the body's inability to produce the enzyme iduronate-2-sulfatase, which is
essential in the continuous process of replacing and breaking down
glycosaminoglycans (GAG). As a result, GAG remains stored in cells in the
body causing progressive damage. The symptoms of Hunter syndrome are usually
not visible at birth, but usually start to become noticeable after the first
year of life. Often the first symptoms may include hernias, frequent ear
infections, runny noses, and abnormal facial appearance. As the disease
progresses, a variety of symptoms appear including, enlarged liver and spleen,
heart failure, obstructive airway disease, sleep apnea, joint stiffness, and,
in the severe form, central nervous system involvement. In severe cases, the
life expectancy for patients with Hunter syndrome is typically 10-15 years of
age. However, in the attenuated form of the disease, patients can survive
into the fifth or sixth decade of life. TKT believes there are up to 2,000
patients worldwide afflicted with Hunter syndrome in jurisdictions where
reimbursement may be possible.
About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company primarily
focused on researching, developing and commercializing treatments for rare
diseases caused by protein deficiencies. Within this focus, the company
markets Replagal(TM), an enzyme replacement therapy for Fabry disease, and is
developing treatments for Hunter syndrome and Gaucher disease. In addition to
its focus on rare diseases, TKT intends to commercialize Dynepo(TM), its Gene-
Activated(R) erythropoietin product for anemia related to kidney disease, in
the European Union. TKT was founded in 1988 and is headquartered in
Cambridge, Massachusetts, with additional operations in Europe, Canada and
South America. Additional information about TKT is available on the company's
website at http://www.tktx.com.
This press release contains forward-looking statements regarding I2S for
Hunter syndrome, as well as statements containing the words "believes,"
"anticipates," "plans," "expects," "estimates," "intends," "should," "could,"
"will," "may," and similar expressions. There are a number of important
factors that could cause the company's actual results to differ materially
from those indicated by such forward-looking statements, including, whether
I2S will be safe and effective as a treatment for Hunter syndrome; whether TKT
will complete clinical trials of I2S; whether the trial described in this
release will be sufficient for regulatory approval or additional studies will
be required; whether the FDA or equivalent authorities will approval I2S on a
timely basis, or at all; whether if approved, this product will achieve
commercial success; whether competing products will reduce any market
opportunity that may exist for I2S; and other factors set forth under the
caption "Certain Factors That May Affect Future Results" in the company's
Quarterly Report on Form 10-Q filed August 9, 2004 with the Securities and
Exchange Commission and are incorporated herein by reference. While the
company may elect to update forward-looking statements at some point in the
future, the company specifically disclaims any obligation to do so, even if
its expectations change.
Gene-Activated(R) is a registered trademark and Replagal(TM) is a
trademark of Transkaryotic Therapies, Inc. Dynepo(TM) is a trademark of
Sanofi-Aventis SA.
For More Information Contact:
Justine E. Koenigsberg
Senior Director, Corporate Communications
(617) 349-0271
Daniella M. Lutz
Corporate Communications Manager
(617) 349-0205
SOURCE Transkaryotic Therapies, Inc.
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http://www.tktx.com
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CONTACT:
Justine E. Koenigsberg, Senior Director,
Corporate Communications, +1-617-349-0271, or Daniella M. Lutz,
Corporate Communications Manager, +1-617-349-0205 both of
Transkaryotic Therapies, Inc.
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